Mancuso Katia, Barbato Simona, Talarico Marco, Tacchetti Paola, Zamagni Elena, Cavo Michele
IRCCS Azienda Ospedaliero-Universitaria di Bologna, Istituto di Ematologia "Seràgnoli", Bologna, Italy.
Dipartimento di Scienze Mediche e Chirurgiche, Università di Bologna, Bologna, Italy.
Expert Opin Biol Ther. 2025 Jan;25(1):27-46. doi: 10.1080/14712598.2024.2433518. Epub 2024 Dec 9.
Modern anti-myeloma therapies have broken new ground in the treatment of the disease, and the incorporation of ide-cel in the treatment landscape represents one of the major scientific and clinical advances.
Ide-cel was the first cell-based gene therapy approved for the treatment of triple-class exposed relapsed/refractory myeloma patients, showing impressive results, and demonstrating superiority over standard regimens in terms of efficacy, potential treatment-free intervals, and improved quality of life in heavily pretreated patients and in high-risk disease. This review summarizes the state-of-the-art of the most recent updates deriving from the use of ide-cel within ongoing, or upcoming, clinical trials, and from real-life experiences.
As the use of chimeric antigen receptor (CAR)-T therapy is likely to progressively increase over time and current indications expand to earlier treatment lines, efforts should be directed toward ameliorating overall management to facilitate proactive planning for treatment sequencing and provide adequate time for logistical planning. Importantly, the potential limited availability of CAR-T therapy highlights the importance of careful patient selection and coordination among centers. Meanwhile, attempts are underway to improve tolerance and reduce toxicity while enhancing anti-myeloma activity.
现代抗骨髓瘤疗法在该疾病的治疗方面取得了新突破,ide-cel纳入治疗领域是主要的科学和临床进展之一。
Ide-cel是首个获批用于治疗接受过三类药物治疗的复发/难治性骨髓瘤患者的基于细胞的基因疗法,显示出令人瞩目的效果,并在疗效、潜在无治疗间期以及改善接受过大量预处理患者和高危疾病患者的生活质量方面优于标准方案。本综述总结了在正在进行或即将开展的临床试验中以及实际临床经验中使用ide-cel所获得的最新进展。
随着嵌合抗原受体(CAR)-T疗法的使用可能会随着时间的推移而逐渐增加,且当前适应症扩展到更早的治疗线,应致力于改善整体管理,以促进积极的治疗顺序规划,并为后勤规划提供足够时间。重要的是,CAR-T疗法潜在的有限可及性凸显了谨慎选择患者以及各中心之间协调的重要性。与此同时,正在尝试提高耐受性、降低毒性,同时增强抗骨髓瘤活性。